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Boston, Mass., Oct. 3, 2013 – In a paper in Cell Stem Cell, a team led by researchers in the Boston Children's Hospital's Stem Cell Transplantation Program reports a new approach for turning induced pluripotent stem cells (iPSCs) into hematopoietic stem and progenitor cells for in vivo disease modeling.

With this strategy—which they call re-specification—the team, including Sergei Doulatov, PhD, and George Daley, MD, PhD, of Boston Children’s, may have overcome technical barriers to generating blood disease-specific animal models from the thousands of iPSC cell lines now sitting in laboratory freezers around the world.

The main advantage of the technique lies in the raw material. The research team started with iPSCs that had already been directed to grow into myeloid progenitors, which are more closely related to the desired blood progenitors than skin or other fully differentiated cell types commonly used in stem cell experiments.

The researchers then used a select set of transcription factors to turn back the molecular clock just a little on these committed myeloid cells, turning them into blood progenitors that readily engrafted and differentiated when transplanted into mice.

The re-specification technique could help generate the large number of engraftable cells needed to create animal models from iPSCs generated from human patients suffering a range of blood disorders, such as anemias, thalassemia or sickle cell disease.

The study was supported by the National Institute of Diabetes and Digestive and Kidney Diseases, the National Heart, Lung and Blood Institute, Alex's Lemonade Stand Foundation for Childhood Cancer, Doris Duke Medical Foundation, the Helen Hay Whitney Foundation and the Howard Hughes Medical Institute.

Boston Children's Hospital is home to the world’s largest research enterprise based at a pediatric medical center, where its discoveries have benefited both children and adults since 1869. More than 1,100 scientists, including seven members of the National Academy of Sciences, 13 members of the Institute of Medicine and 14 members of the Howard Hughes Medical Institute comprise Boston Children’s research community. Founded as a 20-bed hospital for children, Boston Children’s today is a 395-bed comprehensive center for pediatric and adolescent health care grounded in the values of excellence in patient care and sensitivity to the complex needs and diversity of children and families. Boston Children’s is also the primary pediatric teaching affiliate of Harvard Medical School. For more information about research and clinical innovation at Boston Children’s, visit: http://vectorblog.org.

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